Clinical trial design for cutaneous neurofibromas

Ashley Cannon, Kurt Jarnagin, Bruce Korf, Brigitte C. Widemann, Denise Casey, Hon Sum Ko, Jaishri Blakeley, Sharad Verma, Dominique C. Pichard

Research output: Contribution to journalArticle

Abstract

OBJECTIVE: Several clinical trials targeting cutaneous neurofibromas (cNF) have been conducted; however, none has resulted in meaningful changes to care. The Clinical Trial Design and Development subgroup's goals were to (1) define key considerations in the design of clinical trials for cNF, (2) summarize existing data in relation to these considerations, and (3) provide consensus recommendations about key elements of trial design to accelerate the clinical development of therapies for cNF. METHODS: The subgroup, with experts from genetics, dermatology, neurology, oncology, and basic science, spanning academia, government research, and regulatory programs, and industry, reviewed published and unpublished data on clinical trials for cNF and other diseases in the skin. Discussions of these data resulted in formulation of a list of priority issues to address in order to develop efficient and effective clinical trials for cNF. RESULTS: The subgroup identified 2 natural history studies of cNF, 4 priority outcome measures, and 6 patient-reported outcome tools for potential use in efficacy trials of cNF. Time to initiate intervention, patient eligibility, mechanism of action, route of administration, safety monitoring, and regulatory agency interactions were identified as key factors to consider when designing clinical trials for cNF. CONCLUSIONS: Alignment on endpoints and methods for the measurement and quantification of cNF represent a priority for therapeutic development for cNF. Advances in technological methods and outcome tools utilized in other skin diseases may be applicable to cNF studies. Patient age is an important factor guiding trial design and clinical development path.

Original languageEnglish (US)
Pages (from-to)S31-S37
JournalNeurology
Volume91
Issue number2
DOIs
StatePublished - Jul 10 2018

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Neurofibroma
Clinical Trials
Skin
Skin Diseases
Critical Pathways
Neurology
Dermatology
Natural History
Industry
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Cannon, A., Jarnagin, K., Korf, B., Widemann, B. C., Casey, D., Ko, H. S., ... Pichard, D. C. (2018). Clinical trial design for cutaneous neurofibromas. Neurology, 91(2), S31-S37. https://doi.org/10.1212/WNL.0000000000005790

Clinical trial design for cutaneous neurofibromas. / Cannon, Ashley; Jarnagin, Kurt; Korf, Bruce; Widemann, Brigitte C.; Casey, Denise; Ko, Hon Sum; Blakeley, Jaishri; Verma, Sharad; Pichard, Dominique C.

In: Neurology, Vol. 91, No. 2, 10.07.2018, p. S31-S37.

Research output: Contribution to journalArticle

Cannon, A, Jarnagin, K, Korf, B, Widemann, BC, Casey, D, Ko, HS, Blakeley, J, Verma, S & Pichard, DC 2018, 'Clinical trial design for cutaneous neurofibromas', Neurology, vol. 91, no. 2, pp. S31-S37. https://doi.org/10.1212/WNL.0000000000005790
Cannon A, Jarnagin K, Korf B, Widemann BC, Casey D, Ko HS et al. Clinical trial design for cutaneous neurofibromas. Neurology. 2018 Jul 10;91(2):S31-S37. https://doi.org/10.1212/WNL.0000000000005790
Cannon, Ashley ; Jarnagin, Kurt ; Korf, Bruce ; Widemann, Brigitte C. ; Casey, Denise ; Ko, Hon Sum ; Blakeley, Jaishri ; Verma, Sharad ; Pichard, Dominique C. / Clinical trial design for cutaneous neurofibromas. In: Neurology. 2018 ; Vol. 91, No. 2. pp. S31-S37.
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